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Sponsored Artificial Intelligence: A Modern Approach, Global Edition Artificial Intelligence: A Modern Approach, Global Edition

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C. Kleinschnitz

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Open access Sep 2026

Serum circulating cell-free messenger RNA profile response to risdiplam treatment in adult patients with late-onset spinal muscular atrophy

Abstract SMN-enhancing therapies have improved outcomes in adult late-onset spinal muscular atrophy (loSMA), but minimally invasive biomarkers that reflect systemic disease and treatment effects are lacking. In this retrospective cohort study, we profiled circulating cell-free messenger RNA (ccfmRNA) in serum samples f...

M. Leo, Linda-Isabell Schmitt, K. C. Liebig et al. · 0 citations

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